Trial Outcomes & Findings for Over Active Bladder Instillation Study - Botox (NCT NCT00667095)

NCT ID: NCT00667095

Last Updated: 2014-07-11

Results Overview

The I-QoL measures the effect of urinary incontinence on quality of life. It is divided into 3 subscales: 1) avoidance and limiting behavior, 2) psychosocial impact, and 3) social embarrassment. The I-QOL is comprised of 22 items, each with the response scale from '1= Extremely' to '5= Not at all'. A mean score for each subscale is calculated (averaging the scores for the items in each subscale) as well as a total score for all 22 items (sum of all subscale scores). The scores are then transformed to a 'Scale score' ranging from 0-100 points for ease of interpretation: Scale score = (sum of the items - lowest possible score)/possible raw score range X 100. Higher scores indicate less impact of incontinence on quality of life.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

25 participants

Primary outcome timeframe

Baseline, 1 month, 3 months

Results posted on

2014-07-11

Participant Flow

A total of 25 subjects were recruited from 10/21/2008 to 8/19/2010. Three of these were determined to be screen failures before randomization, and another one was withdrawn prior to randomization. Twenty one subjects were randomized: 11 to Botox/DMSO, and 10 to DMSO only.

Participant milestones

Participant milestones
Measure
Botox and DMSO Instillation
Botulinum-A Toxin (Botox) 300 units in 50 cubic centimeters of Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution Botox Instillation: Botox instilled into the bladder via ureteral catheter and retained for up to 30 minutes then spontaneously voided. DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
DMSO Instillation
Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution, 50 cubic centimeters DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
Overall Study
STARTED
11
10
Overall Study
COMPLETED
11
9
Overall Study
NOT COMPLETED
0
1

Reasons for withdrawal

Reasons for withdrawal
Measure
Botox and DMSO Instillation
Botulinum-A Toxin (Botox) 300 units in 50 cubic centimeters of Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution Botox Instillation: Botox instilled into the bladder via ureteral catheter and retained for up to 30 minutes then spontaneously voided. DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
DMSO Instillation
Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution, 50 cubic centimeters DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
Overall Study
Withdrawal by Subject
0
1

Baseline Characteristics

Over Active Bladder Instillation Study - Botox

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Botox and DMSO Instillation
n=11 Participants
Botulinum-A Toxin (Botox) 300 units in 50 cubic centimeters of Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution Botox Instillation: Botox instilled into the bladder via ureteral catheter and retained for up to 30 minutes then spontaneously voided. DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
DMSO Instillation
n=10 Participants
Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution, 50 cubic centimeters DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
Total
n=21 Participants
Total of all reporting groups
Age, Continuous
58 years
n=5 Participants
71 years
n=7 Participants
68 years
n=5 Participants
Sex: Female, Male
Female
11 Participants
n=5 Participants
10 Participants
n=7 Participants
21 Participants
n=5 Participants
Sex: Female, Male
Male
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Region of Enrollment
United States
11 participants
n=5 Participants
10 participants
n=7 Participants
21 participants
n=5 Participants
Smoking Status
Never smoked
4 participants
n=5 Participants
6 participants
n=7 Participants
10 participants
n=5 Participants
Smoking Status
Past smoker
6 participants
n=5 Participants
4 participants
n=7 Participants
10 participants
n=5 Participants
Smoking Status
Current smoker
1 participants
n=5 Participants
0 participants
n=7 Participants
1 participants
n=5 Participants
History of Hypertension
Hypertension
6 participants
n=5 Participants
7 participants
n=7 Participants
13 participants
n=5 Participants
History of Hypertension
No hypertension
5 participants
n=5 Participants
3 participants
n=7 Participants
8 participants
n=5 Participants
History of Urinary Tract Infections (UTIs)
Previous history of UTIs
10 participants
n=5 Participants
10 participants
n=7 Participants
20 participants
n=5 Participants
History of Urinary Tract Infections (UTIs)
No previous UTIs
1 participants
n=5 Participants
0 participants
n=7 Participants
1 participants
n=5 Participants
Number of Vaginal Births
0 vaginal births
3 participants
n=5 Participants
3 participants
n=7 Participants
6 participants
n=5 Participants
Number of Vaginal Births
1-2 vaginal births
4 participants
n=5 Participants
1 participants
n=7 Participants
5 participants
n=5 Participants
Number of Vaginal Births
≥ 3 vaginal births
4 participants
n=5 Participants
6 participants
n=7 Participants
10 participants
n=5 Participants
Body Mass Index
26 kg/m^2
n=5 Participants
30 kg/m^2
n=7 Participants
27 kg/m^2
n=5 Participants
Post-Void Residual Urine
15 mL
n=5 Participants
29 mL
n=7 Participants
17 mL
n=5 Participants
Incontinent Episodes during 24 Hours
6 Episodes
n=5 Participants
3 Episodes
n=7 Participants
4 Episodes
n=5 Participants
Median 24 Hour Pad Weight
166 g
n=5 Participants
179 g
n=7 Participants
166 g
n=5 Participants
Median Number of Pads Per Day
4 pads
n=5 Participants
5 pads
n=7 Participants
5 pads
n=5 Participants

PRIMARY outcome

Timeframe: Baseline, 1 month, 3 months

Population: Analysis was done according to the principle of intention-to-treat. The sample size was reduced to 9 in the Dimethyl Sulfoxide (DMSO) instillation arm at 3 months due to withdrawal for reasons unrelated to study (cancer).

The I-QoL measures the effect of urinary incontinence on quality of life. It is divided into 3 subscales: 1) avoidance and limiting behavior, 2) psychosocial impact, and 3) social embarrassment. The I-QOL is comprised of 22 items, each with the response scale from '1= Extremely' to '5= Not at all'. A mean score for each subscale is calculated (averaging the scores for the items in each subscale) as well as a total score for all 22 items (sum of all subscale scores). The scores are then transformed to a 'Scale score' ranging from 0-100 points for ease of interpretation: Scale score = (sum of the items - lowest possible score)/possible raw score range X 100. Higher scores indicate less impact of incontinence on quality of life.

Outcome measures

Outcome measures
Measure
Botox and DMSO Instillation
n=11 Participants
Botulinum-A Toxin (Botox) 300 units in 50 cubic centimeters of Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution Botox Instillation: Botox instilled into the bladder via ureteral catheter and retained for up to 30 minutes then spontaneously voided. DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
DMSO Instillation
n=10 Participants
Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution, 50 cubic centimeters DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
Change in Incontinence Quality of Life (I-QoL) Score
Change from baseline to 1 month
9.6 units on a scale
Standard Deviation 13.3
-3.9 units on a scale
Standard Deviation 11.6
Change in Incontinence Quality of Life (I-QoL) Score
Change from baseline to 3 months (n=11, 9)
8.6 units on a scale
Standard Deviation 14.4
-4.0 units on a scale
Standard Deviation 9.3

SECONDARY outcome

Timeframe: baseline, 1 month, 3 months

Population: Analysis was done according to the principle of intention-to-treat. The sample size was reduced to 9 in the Dimethyl Sulfoxide (DSMO) instillation arm at 3 months due to withdrawal for reasons unrelated to study (cancer).

The IIQ-7 measures the effect of urinary incontinence on quality of life. It is comprised of 7 items, each with the response scale from "0=Not at all" to "3=Greatly." The scores can range from 0 to 21; a low score indicates less impact of incontinence on quality of life.

Outcome measures

Outcome measures
Measure
Botox and DMSO Instillation
n=11 Participants
Botulinum-A Toxin (Botox) 300 units in 50 cubic centimeters of Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution Botox Instillation: Botox instilled into the bladder via ureteral catheter and retained for up to 30 minutes then spontaneously voided. DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
DMSO Instillation
n=10 Participants
Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution, 50 cubic centimeters DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
Change in Incontinence Impact Questionnaire Short Form (IIQ-7)
Change from baseline to 1 month
-2.5 units on a scale
Standard Deviation 4.0
2.0 units on a scale
Standard Deviation 5.9
Change in Incontinence Impact Questionnaire Short Form (IIQ-7)
Change from baseline to 3 months (n=11, 9)
-1.8 units on a scale
Standard Deviation 4.2
1.1 units on a scale
Standard Deviation 3.8

SECONDARY outcome

Timeframe: baseline, 1 month, 3 months

Population: Analysis was done according to the principle of intention-to-treat. The sample size was reduced to 9 in the DSMO instillation arm at 3 months due to withdrawal for reasons unrelated to study (cancer).

The ICIQ-SF provides a brief and robust measure to assess the impact of symptoms of incontinence on quality of life and outcome of treatment. The questionnaire has 4 items and the score can range from 0 to 21, with greater values indicating increased severity of symptoms and lower quality of life.

Outcome measures

Outcome measures
Measure
Botox and DMSO Instillation
n=11 Participants
Botulinum-A Toxin (Botox) 300 units in 50 cubic centimeters of Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution Botox Instillation: Botox instilled into the bladder via ureteral catheter and retained for up to 30 minutes then spontaneously voided. DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
DMSO Instillation
n=10 Participants
Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution, 50 cubic centimeters DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
Change in International Consultation on Incontinence Questionnaire - Short Form Score (ICIQ-SF)
Change from baseline to 1 month
-2.4 units on a scale
Standard Deviation 5.8
-0.5 units on a scale
Standard Deviation 4.4
Change in International Consultation on Incontinence Questionnaire - Short Form Score (ICIQ-SF)
Change from baseline to 3 months (n=11, 9)
-2.5 units on a scale
Standard Deviation 5.1
0.8 units on a scale
Standard Deviation 4.5

SECONDARY outcome

Timeframe: baseline, 1 month, 3 months

Population: Analysis was done according to the principle of intention-to-treat. The sample size was reduced to 9 in the DSMO instillation arm at 3 months due to withdrawal for reasons unrelated to study (cancer).

The UDI-6 measures the effect of urinary incontinence on quality of life. It consists of 6 items, each with the response scale from "0=Not at all" to "3=Greatly." The scores can range from 0 to 18; a low score indicates less impact of incontinence on quality of life.

Outcome measures

Outcome measures
Measure
Botox and DMSO Instillation
n=11 Participants
Botulinum-A Toxin (Botox) 300 units in 50 cubic centimeters of Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution Botox Instillation: Botox instilled into the bladder via ureteral catheter and retained for up to 30 minutes then spontaneously voided. DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
DMSO Instillation
n=10 Participants
Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution, 50 cubic centimeters DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
Change in Urogenital Distress Inventory (UDI-6)
Change from baseline to 1 month
-1.9 units on a scale
Standard Deviation 3.7
-0.1 units on a scale
Standard Deviation 1.9
Change in Urogenital Distress Inventory (UDI-6)
Change from baseline to 3 months (n=11, 9)
-0.9 units on a scale
Standard Deviation 3.6
-0.4 units on a scale
Standard Deviation 3.5

SECONDARY outcome

Timeframe: baseline, 1 month, 3 months

Population: Analysis was done according to the principle of intention-to-treat. The sample size was reduced to 9 in the DSMO instillation arm at 3 months due to withdrawal for reasons unrelated to study (cancer).

The Blaivas-Groutz Anti-incontinence scale was used as a measure of urinary incontinence. This scale combines information on the number of incontinent episodes in a 24-hour period, 24-hour pad weights, and a qualitative rating by the patient into a single score ranging from 0 to 6. this score is then used to categorize incontinence as none (0), mild (1-2), moderate (3-4), or severe (5-6).

Outcome measures

Outcome measures
Measure
Botox and DMSO Instillation
n=11 Participants
Botulinum-A Toxin (Botox) 300 units in 50 cubic centimeters of Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution Botox Instillation: Botox instilled into the bladder via ureteral catheter and retained for up to 30 minutes then spontaneously voided. DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
DMSO Instillation
n=10 Participants
Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution, 50 cubic centimeters DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
Number of Participants With Decrease in Blaivas-Groutz Anti-Incontinence Score at 1 Month and 3 Months
1 month
6 participants
4 participants
Number of Participants With Decrease in Blaivas-Groutz Anti-Incontinence Score at 1 Month and 3 Months
3 months (n=11, 9)
6 participants
2 participants

SECONDARY outcome

Timeframe: Baseline, 1 month, 3 months

Population: Analysis was done according to the principle of intention-to-treat. The sample size was reduced to 9 in the DSMO instillation arm at 3 months due to withdrawal for reasons unrelated to study (cancer).

Urinary urgency was measured by the Indevus Urgency Severity Scale (IUSS). The IUSS asks patients to assess the severity of 'urgency' at each void. The scale employs the following wording: "Degree of urgency is meant to describe your urge to urinate. Sometimes you may feel a very strong urge to urinate and at other times, you may feel a milder urge prior to the onset of a toilet void. Rate this feeling by circling 0, 1, 2, or 3, defined as: 0: NONE - no urgency, 1: MILD - awareness of urgency, but it is easily tolerated and you can continue with your usual activity or tasks, 2: MODERATE - enough urgency discomfort that it interferes with or shortens your usual activity or tasks, 3: SEVERE - extreme urgency discomfort that abruptly stops all activity or tasks."

Outcome measures

Outcome measures
Measure
Botox and DMSO Instillation
n=11 Participants
Botulinum-A Toxin (Botox) 300 units in 50 cubic centimeters of Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution Botox Instillation: Botox instilled into the bladder via ureteral catheter and retained for up to 30 minutes then spontaneously voided. DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
DMSO Instillation
n=10 Participants
Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution, 50 cubic centimeters DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
Number of Participants With a Decrease in Urinary Urgency at 1 Month and 3 Months
1 month
4 participants
1 participants
Number of Participants With a Decrease in Urinary Urgency at 1 Month and 3 Months
3 months (n=11, 9)
5 participants
0 participants

Adverse Events

Botox and DMSO Instillation

Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths

DMSO Instillation

Serious events: 2 serious events
Other events: 7 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Botox and DMSO Instillation
n=11 participants at risk
Botulinum-A Toxin (Botox) 300 units in 50 cubic centimeters of Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution Botox Instillation: Botox instilled into the bladder via ureteral catheter and retained for up to 30 minutes then spontaneously voided. DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
DMSO Instillation
n=10 participants at risk
Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution, 50 cubic centimeters DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
Nervous system disorders
Hospitalization for body jerking and termons due to oligodendroglioma.
0.00%
0/11
10.0%
1/10 • Number of events 1
Infections and infestations
Hospitalization for joint pain due to infection
0.00%
0/11
10.0%
1/10 • Number of events 1
Infections and infestations
Hospitalization for tibial ulcer
0.00%
0/11
10.0%
1/10 • Number of events 1

Other adverse events

Other adverse events
Measure
Botox and DMSO Instillation
n=11 participants at risk
Botulinum-A Toxin (Botox) 300 units in 50 cubic centimeters of Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution Botox Instillation: Botox instilled into the bladder via ureteral catheter and retained for up to 30 minutes then spontaneously voided. DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
DMSO Instillation
n=10 participants at risk
Dimethyl Sulfoxide (DMSO) 50% w/w aqueous solution, 50 cubic centimeters DMSO Instillation: DMSO 50% w/w aqueous solution; 50 cubic centimeters instilled into the bladder via ureteral catheter and retained up to 30 minutes then spontaneously voided.
Infections and infestations
Urinary Tract Infection
27.3%
3/11 • Number of events 4
50.0%
5/10 • Number of events 6
Cardiac disorders
High Blood Pressue
9.1%
1/11 • Number of events 1
0.00%
0/10
Skin and subcutaneous tissue disorders
Rash
9.1%
1/11 • Number of events 1
0.00%
0/10
Injury, poisoning and procedural complications
Had fall, unable to walk
9.1%
1/11 • Number of events 1
0.00%
0/10
Renal and urinary disorders
Bladder spasms after instillation
0.00%
0/11
10.0%
1/10 • Number of events 1
Blood and lymphatic system disorders
Swollen ankles
0.00%
0/11
10.0%
1/10 • Number of events 1
Blood and lymphatic system disorders
Nose Bleed
0.00%
0/11
10.0%
1/10 • Number of events 1
Injury, poisoning and procedural complications
Left Distal Radius Fracture from a Twist and Fall
0.00%
0/11
10.0%
1/10 • Number of events 1

Additional Information

Dr. Steven P. Petrou

Mayo Clinic

Phone: 904-953-7330

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place